Treatment of BRAF ( B-Rapidly Accelerated Fibrosarcoma) Mutated Papillary Craniopharyngioma

Launched by EVA MARIE ERFURTH, MD, PHD · Aug 30, 2022

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment for a type of brain tumor called papillary craniopharyngioma that has a specific genetic mutation known as BRAF. Participants in the trial will receive two oral medications, dabrafenib and trametinib, which are designed to target and inhibit the effects of the BRAF mutation. The goal is to see how well these medications can reduce the size of the tumor and improve the patients' overall health and quality of life over the course of one year.

To be eligible for this trial, participants must be adults aged 18 or older who have been diagnosed with papillary craniopharyngioma that has the BRAF V600E mutation. They should either be newly diagnosed or have had a recurrence of the tumor that cannot be treated with surgery due to potential risks. Participants will need to be in good overall health and able to provide informed consent. Throughout the study, doctors will monitor the patients’ brain scans, cognitive function, vision, and overall well-being to assess the treatment's effectiveness. This trial is currently recruiting participants and aims to provide new insights into managing this challenging condition.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 1. Histologically verified papillary craniopharyngioma.
• • 2. BRAF mutated V600E (valine 600 glutamine), verified immunohistochemically and by molecular genetic analysis
• • 3. Newly diagnosed tumor, or recurrence after previous surgery, where surgery is not considered to be able to be performed radically without the risk of serious or permanent sequelae.
• • 4. Age over 18 years
• • 5. Functional status according to ECOG (Eastern Cooperative Oncology Group performance status) 0-2
• 6. Adequate organ function:
• • neutrophils\> 1.5 x 109 platelets\> 100 x 109 creatinine \<1.5 x ULN (upper limit of normal) or creatinine clearance \<45 ml / min bilirubin \<1.5 x ULN ASAT (aspartate aminotransferase) / ALAT (alanine aminotransferase) \<2.5 x ULN
• • 7. Ability to understand and give informed consent.
• • 8. Previous cancer, which does not require current treatment is allowed.
• • 9. The patient agrees to use an adequate method to avoid pregnancy.
• Exclusion Criteria:
• • 1. Ongoing treatment in another drug study or other experimental treatment.
• • 2. Previous treatment with BRAF or MEK inhibitors.
• • 3. Hypersensitivity to study drugs.
• • 4. Ongoing treatment with non-authorized drugs, (strong inducers of CYP2C8 or CYP3A4). If the patient is on unauthorized drugs, they must be discontinued at least 14 days before inclusion.
• • 5. Known cardiovascular disease where treatment with MEK inhibitors is considered inappropriate, eg severe heart failure, prolongation of QT time, uncontrolled arrhythmia, recent (\<6 months) cardiac infarction, uncontrolled hypertension.
• • 6. Active bleeding; intracranial hemorrhage last 4 weeks before inclusion.
• • 7. Thromboembolic disease last 6 months and unstable anticoagulant treatment less than 4 weeks before inclusion.
• • 8. Women who are pregnant or breastfeeding.
• • 9. Previous central serous retinopathy or retinal vein occlusion.
• • 10. Previous uveitis or iritis last 4 weeks before inclusion.
• • 11. Surgery within the last 3 weeks.
• • 12. For postoperative patients; radiation therapy within the last 3 months.